Pluto Bioinformatics

GSE148529: bT87Q-globin gene therapy reduces sickle hemoglobin production, allowing for ex vivo anti-sickling activity in human erythroid cells

Bulk RNA sequencing

We performed RNA-seq analysis on immortalized human erythroid cell line (HUDEP-2), that had been engineered to carry the sickle cell disease (SCD) mutation (sHUDEP-2), and on healthy donor mobilized CD34+ derived cells after transduction with a control GFP lentivirus and lentivirus expressing T87Q-globin. Our results show activation of inflammation- and proliferation-related programs, suggesting minimal changes of background gene expression except for T87Q-globin expression and endogenous /s-globin suppression. SOURCE: Mehdi Pirooznia (mehdi.pirooznia@nih.gov) - National Institutes of Health